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CompletedNCT03213678Updated Apr 21, 2026Results posted

Samotolisib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With TSC or PI3K/MTOR Mutations (A Pediatric MATCH Treatment Trial)

A Phase 2 interventional study of Biospecimen Collection and Computed Tomography in Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Non-Hodgkin Lymphoma and Ann Arbor Stage IV Non-Hodgkin Lymphoma, sponsored by National Cancer Institute (NCI). Completed at 127 sites in 2 countries. Open to participants aged 12 Months to 21 Years. Per ClinicalTrials.gov, last updated 2026-04-21.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
18
Allocation
Not applicable
Ages
12 Months to 21 Years
Sex
All
01

Study summary

This phase II Pediatric MATCH trial studies how well samotolisib works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with TSC or PI3K/MTOR mutations that have spread to other places in the body (metastatic) and have come back (recurrent) or do not respond to treatment (refractory). Samotolisib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Read the detailed description

PRIMARY OBJECTIVE:

I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with samotolisib (LY3023414) with advanced solid tumors, non-Hodgkin lymphomas or central nervous system (CNS) tumors that harbor TSC loss of function mutations, and/or other PI3K/MTOR activating mutations.

SECONDARY OBJECTIVES:

I. To estimate the progression free survival in pediatric patients treated with LY3023414 with advanced solid tumors, non-Hodgkin lymphomas or CNS tumors that harbor TSC loss of function mutations, and/or other PI3K/MTOR activating mutations.

II. To obtain information about the tolerability of LY3023414 in children with relapsed or refractory cancer.

III. To characterize the pharmacokinetics of LY3023414 in children with recurrent or refractory cancer.

EXPLORATORY OBJECTIVES:

I. To increase knowledge of the genomic landscape of relapsed pediatric solid tumors and lymphomas and identify potential predictive biomarkers (other than the genomic alteration for which study treatment was assigned) using additional genomic, transcriptomic, and proteomic testing platforms.

II. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA).

III. To evaluate the frequency and mechanism of biallelic loss of function, and evaluate the expression of TSC1, TSC2, and PTEN in subjects who enroll with a loss of function mutation in one of these genes.

OUTLINE: This is a dose-escalation study.

Patients receive samotolisib orally (PO) twice daily (BID) on days 1-28. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unexpected toxicity. Patients undergo an x-ray, computed tomography (CT), magnetic resonance imaging (MRI), fludeoxyglucose F-18 (FDG)-position emission tomography (FDG-PET), and blood sample collection on study.

After completion of study treatment, patients are followed up periodically.

02

Conditions studied

  • Advanced Malignant Solid Neoplasm
  • Ann Arbor Stage III Non-Hodgkin Lymphoma
  • Ann Arbor Stage IV Non-Hodgkin Lymphoma
  • Malignant Glioma
  • Recurrent Ependymoma
  • Recurrent Ewing Sarcoma
  • Recurrent Glioma
  • Recurrent Hepatoblastoma
  • Recurrent Langerhans Cell Histiocytosis
  • Recurrent Malignant Germ Cell Tumor
  • Recurrent Malignant Solid Neoplasm
  • Recurrent Medulloblastoma
  • Recurrent Neuroblastoma
  • Recurrent Non-Hodgkin Lymphoma
  • Recurrent Osteosarcoma
  • Recurrent Peripheral Primitive Neuroectodermal Tumor
  • Recurrent Primary Central Nervous System Neoplasm
  • Recurrent Rhabdomyosarcoma
  • Recurrent Soft Tissue Sarcoma
  • Refractory Langerhans Cell Histiocytosis
  • Refractory Malignant Germ Cell Tumor
  • Refractory Malignant Solid Neoplasm
  • Refractory Neuroblastoma
  • Refractory Non-Hodgkin Lymphoma
  • Refractory Primary Central Nervous System Neoplasm
  • Rhabdoid Tumor
  • Stage III Osteosarcoma AJCC v7
  • Stage III Soft Tissue Sarcoma AJCC v7
  • Stage IV Osteosarcoma AJCC v7
  • Stage IV Soft Tissue Sarcoma AJCC v7
  • Stage IVA Osteosarcoma AJCC v7
  • Stage IVB Osteosarcoma AJCC v7
  • Wilms Tumor
03

In context

Glioma

1,452 studies on the registry are indexed under Glioma; 349 are open to participants now.

This study's enrollment of 18 is below the median of 32 across 1,075 interventional studies indexed under Glioma.

Browse Glioma studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Browse National Cancer Institute (NCI) studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Months to 21 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Patient must have enrolled onto APEC1621SC/NCI-2017-01251/ NCT03155620 and must have been given a treatment assignment to Molecular Analysis for Therapy Choice (MATCH) to APEC1621D based on the presence of an actionable mutation as defined in APEC1621SC; note that treatment assignment may be to primary cohort A for patients with TSC1 or TSC2 loss of function mutations or primary cohort B for patients with other PI3K/MTOR pathway mutations
  • Patients accruing to dose level 1 must have a body surface area >= 0.52 m\^2 at the time of study enrollment; patients accruing to dose level 2 must have a body surface area >= 0.37 m\^2 at the time of study enrollment; patients accruing to dose level -1 must have a body surface area >= 0.75 m\^2 at the time of study enrollment

  • Patients must have radiographically at the time of study enrollment; patients with neuroblastoma who do not have measurable disease but have metaiodobenzylguanidine (MIBG) positive (+) evaluable disease are eligible; measurable disease in patients with CNS involvement is defined as any lesion that is at minimum 10 mm in one dimension on standard magnetic resonance imaging (MRI) or computed tomography (CT)

    • Note: The following do not qualify as measurable disease:

      • Malignant fluid collections (e.g., ascites, pleural effusions)
      • Bone marrow infiltration except that detected by MIBG scan for neuroblastoma
      • Lesions only detected by nuclear medicine studies (e.g., bone, gallium or positron emission tomography [PET] scans) except as noted for neuroblastoma
      • Elevated tumor markers in plasma or cerebrospinal fluid (CSF)
      • Previously radiated lesions that have not demonstrated clear progression post radiation
      • lesions that do not meet the measurement requirements for (RECIST) 1.1
      • Bone lesions without an associated soft tissue mass >= 10 mm in greatest diameter; bone lesions with an associated soft tissue mass >= 10 mm in greatest diameter imaged by CT or MRI are considered measurable
  • >= 50% for patients > 16 years of age and Lansky >= 50 for patients =\< 16 years of age; Note: neurologic deficits in patients with CNS tumors must have been stable for at least 7 days prior to study enrollment; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score

  • Patients must have fully recovered from the acute toxic effects of all prior anti-cancer therapy and must meet the following minimum duration from prior anti-cancer directed therapy prior to enrollment; if after the required timeframe, the numerical eligibility criteria are met, e.g. blood count criteria, the patient is considered to have recovered adequately

    • Cytotoxic chemotherapy or other anti-cancer agents known to be myelosuppressive; >= 21 days after the last dose of cytotoxic or myelosuppressive chemotherapy (42 days if prior nitrosourea)
    • Anti-cancer agents not known to be myelosuppressive (e.g. not associated with reduced or [ANC] counts): >= 7 days after the last dose of agent
    • Antibodies: >= 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to grade =\< 1
    • : if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
    • Hematopoietic growth factors: >= 14 days after the last dose of a long-acting growth factor (e.g. pegfilgrastim) or 7 days for short-acting growth factor; for growth factors that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur; the duration of this interval must be discussed with the study chair and the study-assigned research coordinator
    • Interleukins, interferons and cytokines (other than hematopoietic growth factors): >= 21 days after the completion of interleukins, interferon or cytokines (other than hematopoietic growth factors)

    • Stem cell Infusions (with or without total body irradiation [TBI]):

      • Allogeneic (non-autologous) bone marrow or stem cell transplant, or any stem cell infusion including donor lymphocyte infusion (DLI) or boost infusion: >= 84 days after infusion and no evidence of (GVHD)
      • Autologous stem cell infusion including boost infusion: >= 42 days
    • Cellular therapy: >= 42 days after the completion of any type of cellular therapy (e.g. modified T cells, natural killer [NK] cells, dendritic cells, etc.)

    • Radiation therapy (XRT)/external beam irradiation including protons: >= 14 days after local XRT; >= 150 days after TBI, craniospinal XRT or if radiation to >= 50% of the pelvis; >= 42 days if other substantial bone marrow (BM) radiation

      • Note: radiation may not be delivered to "measurable disease" tumor site(s) being used to follow response to subprotocol treatment
    • Radiopharmaceutical therapy (e.g., radiolabeled antibody, iobenguane I-131 [131I-MIBG]): >= 42 days after systemically administered radiopharmaceutical therapy
    • Patients must not have received prior exposure to LY3023414
    • Patients must not have received prior exposure to an agent specifically directed at the PI3K/MTOR pathway (a PI3K inhibitor, an AKT inhibitor, an MTOR inhibitor, including rapalogs, or a combined PI3K/MTOR inhibitor)

  • For patients with solid tumors without known bone marrow involvement:

    • Peripheral absolute neutrophil count (ANC) >= 1000/mm\^3
    • Platelet count >= 100,000/mm\^3 (transfusion independent, defined as not receiving platelet transfusions for at least 7 days prior to enrollment)
  • Patients with known bone marrow disease will be eligible for study provided they meet the blood counts (may receive transfusions provided they are not known to be to red cell or platelet transfusions); these patients will not be evaluable for hematologic toxicity

  • or radioisotope (GFR) >= 70 ml/min/1.73 m\^2 or a serum creatinine based on age/gender as follows:

    • Age: 1 to \< 2 years; maximum serum creatinine (mg/dL): male 0.6; female 0.6
    • Age: 2 to \< 6 years; maximum serum creatinine (mg/dL): male 0.8; female 0.8
    • Age: 6 to \< 10 years; maximum serum creatinine (mg/dL): male 1; female 1
    • Age: 10 to \< 13 years; maximum serum creatinine (mg/dL): male 1.2; female 1.2
    • Age: 13 to \< 16 years; maximum serum creatinine (mg/dL): male 1.5; female 1.4
    • Age: >= 16 years; maximum serum creatinine (mg/dL): male 1.7; female 1.4
  • (sum of conjugated + unconjugated) =\< 1.5 x (ULN) for age
  • Serum glutamate pyruvate transaminase () (alanine aminotransferase [ALT]) =\< 135 U/L; (for the purpose of this study, the ULN for SGPT is 45 U/L)
  • Serum albumin >= 2 g/dL
  • Patients must have a normal blood sugar level for age; if an initial random draw (i.e. non-fasting) blood glucose value is out of range, it is acceptable to repeat this test as a fasting draw
  • Patients must have a serum triglyceride level =\< 300 mg/dL and serum cholesterol level =\< 300 mg/dL; if an initial random draw (i.e. non-fasting) is out of range, it is acceptable to repeat this test as a fasting draw
  • Patients with seizure disorder may be enrolled if on anticonvulsants and well controlled
  • Nervous system disorders (by version 5.0 [CTCAE V 5.0]) resulting from prior therapy must be =\< grade 2, with the exception of decreased tendon reflex (DTR); any grade of DTR is eligible
  • (QTc) interval =\< 480 milliseconds
  • Patients must be able to swallow intact tablets
  • All patients and/or their parents or legally authorized representatives must sign a written ; assent, when appropriate, will be obtained according to institutional guidelines

Exclusion criteria

Exclusion Criteria:

  • Pregnant or breast-feeding women will not be entered on this study; pregnancy tests must be obtained in girls who are post-menarchal; males or females of may not participate unless they have agreed to use an effective method while receiving study treatment and for 3 months after the last dose of LY3023414

  • Concomitant medications

    • : patients receiving corticosteroids who have not been on a stable or decreasing dose of corticosteroid for at least 7 days prior to enrollment are not eligible; if used to modify immune adverse events related to prior therapy, >= 14 days must have elapsed since last dose of corticosteroid
    • drugs: patients who are currently receiving another investigational drug are not eligible
    • Anti-cancer agents: patients who are currently receiving other anti-cancer agents are not eligible
    • Anti- agents post-transplant: patients who are receiving cyclosporine, tacrolimus or other agents to prevent graft-versus-host disease post bone marrow transplant are not eligible for this trial
  • Patients who have an uncontrolled infection are not eligible
  • Patients who have insulin dependent diabetes are not eligible
  • Patients who have received a prior solid organ transplantation are not eligible
  • Patients with subependymal giant cell astrocytomas (SEGAs) are not eligible
  • Patients who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study are not eligible
Terms in these criteria, in plain language(21)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Measurable disease

The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.

The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.

Leptomeningeal disease

Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.

Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.

RECIST

The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.

“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

Platelets

Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.

Absolute neutrophil count (ANC)

The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.

It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

GVHD (graft-versus-host disease)

After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.

Metastases

Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.

Relapsed and refractory

Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

GFR and eGFR (kidney filtering)

GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².

Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ULN (upper limit of normal)

The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.

“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

CTCAE grade

The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.

1
Mild.
2
Moderate.
3
Severe.
4
Life-threatening.
5
Death due to the event.

“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.

QT and QTc

On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.

The study may name a correction formula and a limit in milliseconds, often around 450–480.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Reproductive potential

Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.

Criteria about it usually ask for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
18 participants (actual)

Study arms

  • Experimental
    Treatment (samotolisib)

    Patients receive samotolisib PO BID on days 1-28. Treatment repeats every 28 days for up to 6 cycles in the absence of disease progression or unexpected toxicity. Patients undergo an x-ray, CT, MRI, FDG-PET, and blood sample collection on study.

    Procedure: Biospecimen Collection · Procedure: Computed Tomography · Procedure: FDG-Positron Emission Tomography · Procedure: Magnetic Resonance Imaging · Drug: Samotolisib · Procedure: X-Ray Imaging

Interventions

  • ProcedureBiospecimen Collection

    Undergo blood specimen collection

    Also known as: Biological Sample Collection, Biospecimen Collected, Specimen Collection

  • ProcedureComputed Tomography

    Undergo CT

    Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, tomography

  • ProcedureFDG-Positron Emission Tomography

    Undergo FDG-PET

    Also known as: FDG, FDG-PET, FDG-PET Imaging

  • ProcedureMagnetic Resonance Imaging

    Undergo MRI

    Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI

  • DrugSamotolisib

    Given PO

    Also known as: 2H-Imidazo(4,5-C)quinolin-2-one, 1,3-Dihydro-8-(5-(1-hydroxy-1-methylethyl)-3-pyridinyl)-1-((2S)-2-methoxypropyl)-3-methyl-, LY 3023414, LY-3023414, LY3023414, WHO 10889

  • ProcedureX-Ray Imaging

    Undergo x-ray

    Also known as: Conventional X-Ray, Diagnostic Radiology, Medical Imaging, X-Ray, Plain film radiographs, Radiographic Imaging, Radiographic imaging procedure (procedure), Radiography, RG, Static X-Ray, X-Ray

06

What researchers measure

Primary outcomes

  1. Objective Response Rate

    A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system (CNS) tumors).

    Time frame: Up to 2 years from study entry

Secondary outcomes

  1. Percentage of Patients Experiencing Grade 3 or 4 Adverse Events

    Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0

    Time frame: Up to 2 years from study entry

  2. Progression Free Survival (PFS)

    The Kaplan-Meier method will be used to estimate the 3-month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact.

    Time frame: Up to 3 months from study entry

  3. Pharmacokinetic (PK) of Samotolisib, Area Under the Curve (AUC).

    The mean (sd) of the AUC

    Time frame: Up to day 15 of cycle 1

Other outcomes

  1. Potential Predictive Biomarker Identification Using Additional Genomic, Transcriptomic, and Proteomic Testing Platforms

    A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

    Time frame: Up to 3 years

  2. Biallelic Loss of Function Frequency and Mechanism

    A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

    Time frame: Up to 3 years

  3. Change in Tumor Genomic Profile

    A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

    Time frame: Baseline up to 3 years

  4. TSC1, TSC2, and PTEN Expression Levels

    A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

    Time frame: Up to 3 years

07

Results

Posted Sep 4, 2024

Participant flow

Participant flow — Overall Study
MilestoneTreatment (Samotolisib)
Started18
Completed0
Not completed18
Withdrew: Death1
Withdrew: Physician decision1
Withdrew: Progressive disease16

Outcome measures

PrimaryObjective Response Rate

A responder is defined as a patient who achieves a best response of partial response or complete response on the study. Response rates will be calculated as the percent of evaluable patients who are responders. The revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) was used to determine response and progression in this study, with specific criteria outlined for the different subtypes of tumors (e.g., 2-dimensional measurements for central nervous system (CNS) tumors).

Time frame:
Up to 2 years from study entry
Reported as:
Number · percentage of patients
Objective Response Rate
percentage of patientsTreatment (Samotolisib)
Objective Response Rate0 (0 to 0)
SecondaryPercentage of Patients Experiencing Grade 3 or 4 Adverse Events

Percentage of patients experiencing grade 3 or higher adverse events will be evaluated according to National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0

Time frame:
Up to 2 years from study entry
Reported as:
Number · Percentage of patients
Percentage of Patients Experiencing Grade 3 or 4 Adverse Events
Percentage of patientsTreatment (Samotolisib)
Percentage of Patients Experiencing Grade 3 or 4 Adverse Events82.4 (56.6 to 96.2)
SecondaryProgression Free Survival (PFS)

The Kaplan-Meier method will be used to estimate the 3-month PFS. PFS is defined as time from initiation of protocol treatment to disease progression, recurrence, death from any cause, or date of last contact.

Time frame:
Up to 3 months from study entry
Reported as:
Number · percentage of participants
Progression Free Survival (PFS)
percentage of participantsTreatment (Samotolisib)
Progression Free Survival (PFS)12 (2 to 31)
SecondaryPharmacokinetic (PK) of Samotolisib, Area Under the Curve (AUC).

The mean (sd) of the AUC

Time frame:
Up to day 15 of cycle 1
Reported as:
Mean · h*μg/mL
Pharmacokinetic (PK) of Samotolisib, Area Under the Curve (AUC).
h*μg/mLTreatment (Samotolisib)
Pharmacokinetic (PK) of Samotolisib, Area Under the Curve (AUC).2323 ± 1006
Other pre-specifiedPotential Predictive Biomarker Identification Using Additional Genomic, Transcriptomic, and Proteomic Testing Platforms

A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

Time frame:
Up to 3 years

Results for this outcome have not been posted.

Other pre-specifiedBiallelic Loss of Function Frequency and Mechanism

A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

Time frame:
Up to 3 years

Results for this outcome have not been posted.

Other pre-specifiedChange in Tumor Genomic Profile

A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

Time frame:
Baseline up to 3 years

Results for this outcome have not been posted.

Other pre-specifiedTSC1, TSC2, and PTEN Expression Levels

A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.

Time frame:
Up to 3 years

Results for this outcome have not been posted.

Adverse events

Collected over Adverse Events monitored/assessed from enrollment to 30 days after the end of treatment, up to 2 years. All-Cause Mortality monitored/assessed up to 5 years. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Treatment (Samotolisib)14/18 (77.8%)9/17 (52.9%)16/17 (94.1%)
Most frequent serious events
Showing 10 of 14
Most frequent serious events
EventTreatment (Samotolisib)
Death NOSGeneral disorders2/17
Lung infectionInfections and infestations2/17
Pain in extremityMusculoskeletal and connective tissue disorders2/17
Heart failureCardiac disorders1/17
Mucositis oralGastrointestinal disorders1/17
Disease progressionGeneral disorders1/17
PainGeneral disorders1/17
Infections and infestations - Other, specifyInfections and infestations1/17
Muscle weakness right-sidedNervous system disorders1/17
HematuriaRenal and urinary disorders1/17
Most frequent other events
Showing 10 of 91
Most frequent other events
EventTreatment (Samotolisib)
FatigueGeneral disorders11/17
AnemiaBlood and lymphatic system disorders9/17
NauseaGastrointestinal disorders9/17
HyperglycemiaMetabolism and nutrition disorders8/17
Alanine aminotransferase increasedInvestigations7/17
Lymphocyte count decreasedInvestigations7/17
HyponatremiaMetabolism and nutrition disorders7/17
ConstipationGastrointestinal disorders6/17
FeverGeneral disorders6/17
White blood cell decreasedInvestigations6/17

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Treatment (Samotolisib)
<=18 years14
Between 18 and 65 years4
>=65 years0
Age, Continuous
Age, Continuous(years)Treatment (Samotolisib)
Mean14.8 ± 4.3
Sex: Female, Male
Sex: Female, Male(Participants)Treatment (Samotolisib)
Female9
Male9
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Treatment (Samotolisib)
Hispanic or Latino4
Not Hispanic or Latino14
Unknown or Not Reported0
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Treatment (Samotolisib)
American Indian or Alaska Native0
Asian0
Native Hawaiian or Other Pacific Islander0
Black or African American2
White14
More than one race0
Unknown or Not Reported2
Region of Enrollment
Region of Enrollment(participants)Treatment (Samotolisib)
United States18
08

Study locations

127 sites
  • Children's Hospital of Alabama
    Birmingham, Alabama 35233, United States
  • Providence Alaska Medical Center
    Anchorage, Alaska 99508, United States
  • Banner Children's at Desert
    Mesa, Arizona 85202, United States
  • Banner University Medical Center - Tucson
    Tucson, Arizona 85719, United States
  • Arkansas Children's Hospital
    Little Rock, Arkansas 72202-3591, United States
  • Kaiser Permanente Downey Medical Center
    Downey, California 90242, United States
  • City of Hope Comprehensive Cancer Center
    Duarte, California 91010, United States
  • Loma Linda University Medical Center
    Loma Linda, California 92354, United States
  • Miller Children's and Women's Hospital Long Beach
    Long Beach, California 90806, United States
  • Children's Hospital Los Angeles
    Los Angeles, California 90027, United States
  • Mattel Children's Hospital UCLA
    Los Angeles, California 90095, United States
  • Valley Children's Hospital
    Madera, California 93636, United States
  • UCSF Benioff Children's Hospital Oakland
    Oakland, California 94609, United States
  • Kaiser Permanente-Oakland
    Oakland, California 94611, United States
  • University of California Davis Comprehensive Cancer Center
    Sacramento, California 95817, United States
  • UCSF Medical Center-Mission Bay
    San Francisco, California 94158, United States
  • Children's Hospital Colorado
    Aurora, Colorado 80045, United States
  • Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
    Denver, Colorado 80218, United States
  • Yale University
    New Haven, Connecticut 06520, United States
  • Alfred I duPont Hospital for Children
    Wilmington, Delaware 19803, United States
  • MedStar Georgetown University Hospital
    Washington D.C., District of Columbia 20007, United States
  • Children's National Medical Center
    Washington D.C., District of Columbia 20010, United States
  • University of Florida Health Science Center - Gainesville
    Gainesville, Florida 32610, United States
  • Nemours Children's Clinic-Jacksonville
    Jacksonville, Florida 32207, United States
  • University of Miami Miller School of Medicine-Sylvester Cancer Center
    Miami, Florida 33136, United States
  • Nicklaus Children's Hospital
    Miami, Florida 33155, United States
  • AdventHealth Orlando
    Orlando, Florida 32803, United States
  • Arnold Palmer Hospital for Children
    Orlando, Florida 32806, United States
  • Nemours Children's Hospital
    Orlando, Florida 32827, United States
  • Nemours Children's Clinic - Pensacola
    Pensacola, Florida 32504, United States
  • Johns Hopkins All Children's Hospital
    St. Petersburg, Florida 33701, United States
  • Saint Joseph's Hospital/Children's Hospital-Tampa
    Tampa, Florida 33607, United States
  • Children's Healthcare of Atlanta - Arthur M Blank Hospital
    Atlanta, Georgia 30329, United States
  • Memorial Health University Medical Center
    Savannah, Georgia 31404, United States
  • Saint Luke's Cancer Institute - Boise
    Boise, Idaho 83712, United States
  • University of Chicago Comprehensive Cancer Center
    Chicago, Illinois 60637, United States
  • Loyola University Medical Center
    Maywood, Illinois 60153, United States
  • Saint Jude Midwest Affiliate
    Peoria, Illinois 61637, United States
  • Southern Illinois University School of Medicine
    Springfield, Illinois 62702, United States
  • Riley Hospital for Children
    Indianapolis, Indiana 46202, United States
  • Blank Children's Hospital
    Des Moines, Iowa 50309, United States
  • University of Iowa/Holden Comprehensive Cancer Center
    Iowa City, Iowa 52242, United States
  • Norton Children's Hospital
    Louisville, Kentucky 40202, United States
  • Children's Hospital New Orleans
    New Orleans, Louisiana 70118, United States
  • Ochsner Medical Center Jefferson
    New Orleans, Louisiana 70121, United States
  • Eastern Maine Medical Center
    Bangor, Maine 04401, United States
  • Sinai Hospital of Baltimore
    Baltimore, Maryland 21215, United States
  • Johns Hopkins University/Sidney Kimmel Cancer Center
    Baltimore, Maryland 21287, United States
  • Massachusetts General Hospital Cancer Center
    Boston, Massachusetts 02114, United States
  • Dana-Farber Cancer Institute
    Boston, Massachusetts 02215, United States
  • C S Mott Children's Hospital
    Ann Arbor, Michigan 48109, United States
  • Michigan State University Clinical Center
    East Lansing, Michigan 48824, United States
  • Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
    Grand Rapids, Michigan 49503, United States
  • Bronson Methodist Hospital
    Kalamazoo, Michigan 49007, United States
  • Children's Hospitals and Clinics of Minnesota - Minneapolis
    Minneapolis, Minnesota 55404, United States
  • University of Minnesota/Masonic Cancer Center
    Minneapolis, Minnesota 55455, United States
  • University of Mississippi Medical Center
    Jackson, Mississippi 39216, United States
  • Children's Mercy Hospitals and Clinics
    Kansas City, Missouri 64108, United States
  • Cardinal Glennon Children's Medical Center
    St Louis, Missouri 63104, United States
  • Washington University School of Medicine
    St Louis, Missouri 63110, United States
  • Mercy Hospital Saint Louis
    St Louis, Missouri 63141, United States
  • Children's Hospital and Medical Center of Omaha
    Omaha, Nebraska 68114, United States
  • University of Nebraska Medical Center
    Omaha, Nebraska 68198, United States
  • University Medical Center of Southern Nevada
    Las Vegas, Nevada 89102, United States
  • Sunrise Hospital and Medical Center
    Las Vegas, Nevada 89109, United States
  • Alliance for Childhood Diseases/Cure 4 the Kids Foundation
    Las Vegas, Nevada 89135, United States
  • Summerlin Hospital Medical Center
    Las Vegas, Nevada 89144, United States
  • Hackensack University Medical Center
    Hackensack, New Jersey 07601, United States
  • Morristown Medical Center
    Morristown, New Jersey 07960, United States
  • Saint Peter's University Hospital
    New Brunswick, New Jersey 08901, United States
  • Albany Medical Center
    Albany, New York 12208, United States
  • Roswell Park Cancer Institute
    Buffalo, New York 14263, United States
  • The Steven and Alexandra Cohen Children's Medical Center of New York
    New Hyde Park, New York 11040, United States
  • NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
    New York, New York 10032, United States
  • Memorial Sloan Kettering Cancer Center
    New York, New York 10065, United States
  • NYP/Weill Cornell Medical Center
    New York, New York 10065, United States
  • University of Rochester
    Rochester, New York 14642, United States
  • State University of New York Upstate Medical University
    Syracuse, New York 13210, United States
  • Montefiore Medical Center - Moses Campus
    The Bronx, New York 10467, United States
  • New York Medical College
    Valhalla, New York 10595, United States
  • Mission Hospital
    Asheville, North Carolina 28801, United States
  • Carolinas Medical Center/Levine Cancer Institute
    Charlotte, North Carolina 28203, United States
  • Novant Health Presbyterian Medical Center
    Charlotte, North Carolina 28204, United States
  • Duke University Medical Center
    Durham, North Carolina 27710, United States
  • Sanford Broadway Medical Center
    Fargo, North Dakota 58122, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, Ohio 45229, United States
  • Rainbow Babies and Childrens Hospital
    Cleveland, Ohio 44106, United States
  • Cleveland Clinic Foundation
    Cleveland, Ohio 44195, United States
  • Nationwide Children's Hospital
    Columbus, Ohio 43205, United States
  • Dayton Children's Hospital
    Dayton, Ohio 45404, United States
  • ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
    Toledo, Ohio 43606, United States
  • University of Oklahoma Health Sciences Center
    Oklahoma City, Oklahoma 73104, United States
  • Legacy Emanuel Children's Hospital
    Portland, Oregon 97227, United States
  • Oregon Health and Science University
    Portland, Oregon 97239, United States
  • Geisinger Medical Center
    Danville, Pennsylvania 17822, United States
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • Children's Hospital of Pittsburgh of UPMC
    Pittsburgh, Pennsylvania 15224, United States
  • Prisma Health Richland Hospital
    Columbia, South Carolina 29203, United States
  • BI-LO Charities Children's Cancer Center
    Greenville, South Carolina 29605, United States
  • Sanford USD Medical Center - Sioux Falls
    Sioux Falls, South Dakota 57117-5134, United States

Showing the first 100 of 127 sites across 2 countries. Choose a country and city above to see the rest.

09

References and documents

Publications

  • Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14. PubMed 31401903 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Nov 2017 to primary completion in Sep 2023, 70 months.

Results were first posted in Sep 2024, 11 months after primary completion.

Of 108 non-industry phase 2 interventional studies under Glioma, half reached primary completion within 55 months of starting (the median), counting those still running; a quarter within 41 and three quarters within 89. This study's 70 months is above that.

Counting every sponsor, half of 131 studies reached it within 54 months; a quarter within 40 and three quarters within 88.

How these figures are worked out, and which studies are left out of them.

Registered
Jul 10, 2017
First posted
Jul 11, 2017
StartActual
Nov 28, 2017
Primary completionActual
Sep 30, 2023
CompletionActual
Dec 31, 2024
Results first posted
Sep 4, 2024
Last update
Apr 21, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Apr 21, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03213678
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Jul 11, 2017
Start date
Nov 28, 2017
Primary completion
Sep 30, 2023
Completion
Dec 31, 2024
Results posted
Sep 4, 2024
Last update
Apr 21, 2026

Study contacts

Theodore W Laetsch
principal investigator · Children's Oncology Group

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: LY3023414

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